ICER Weekly View 04-20-18

From the desk of Mitchell Stein

 

Good morning. A busy week, starting with breakthrough lung cancer news out of American Association of Cancer Researchers conference (see Weekly View in-depth for details), news of an upcoming Presidential speech on drug pricing (See Inside the Beltway), and continuing developments in a potential Shire buyout (Allergan was both in and out on Thursday, see Company/Drug Specific section for details). All that and more below. Before we get started, allow me to suggest you consider ending your week with a 60 Minutes report on Alzheimer’s filmed over the past ten years. Here’s today’s full run-down:

ICER in the news – Final evidence report on Emicizumab, AP includes ICER’s opioid work, and coverage of ICER’s migraine draft report

Inside the Beltway – President to give speech on drug pricing, Gottlieb talks revamped drug review processes, Sharpless talks priorities, and Maryland drug price law struck down

Industry Trends – Imbruvica pricing strategy furor, JAMA piece on cancer drug value, Harvard Pilgrim inks another outcomes deal, China and biotech (2 articles), Amazon takes a step back, and drug disposal kiosks coming to a store near you

Company/Drug Specific – Shire’s future, Sanofi deal, Shire deal (not that one, a different one), Beta Thalassemia gene therapy, cannabis-derived epilepsy drug, and FDA about-face on Alkermes’s depression drug

Weekly View In-depth – Lung cancer, immunotherapy, and checkpoint inhibitors, oh my!

Now, on to the news.


ICER In The News

ICER released a Final Evidence Report and Report-at-a-Glance on emicizumab (Hemlibra®, Genentech) for the prevention of bleeds (prophylaxis) in a subset of individuals with hemophilia A and inhibitors to the clotting protein Factor VIII. ICER’s report was reviewed at a public meeting of the New England Comparative Effectiveness Public Advisory Council (New England CEPAC), where a majority of the Council voted that evidence demonstrates a net health benefit of emicizumab compared to either no prophylaxis or prophylaxis with bypassing agents. The Council also emphasized the drug’s benefits beyond those captured in clinical trials. ICER’s report found that the drug lowers overall costs of treatment for patients with hemophilia A and inhibitors as compared to current therapies.

This week the FDA also tagged emicizumab with the “breakthrough” designation; speeding approval of a broader label indication for the drug. FiercePharma reported on both developments: Roche gets double dose of good Hemlibra news with FDA breakthrough, ICER backing. Additionally, the Pharma Letter covered the ICER release.

Institute for Clinical and Economic Review’s Final Report on Emicizumab Shows New Treatment Improves Health While Lowering Costs for Certain People with Hemophilia A

 

Associated Press story on opioid policy cites ICER’s work on the topic (the story appeared in multiple outlets, below is one example).

Trump builds on Obama opioid policy – KTAR.com

WASHINGTON (AP) — Deep within President Donald Trump’s plan to combat opioid abuse, overshadowed by his call for the death penalty for some drug traffickers, is a push to expand the use of medication to treat addiction.

There was coverage of ICER’s draft report on migraine drugs in BioPharma (below) AJMC.com (ICER Study Compares Cost-Effectiveness of CGRP Inhibitors for Migraine) and in the Pharma Letter (Novartis bests Teva in draft ICER report into CGRP inhibitors subscription required). Also this week, additional study results were announced for Aimovig (one of the drugs in the review): Amgen’s Aimovig halved migraine days in 30 percent of trial patients.

ICER releases draft report on CGRP migraine drugs

The Institute for Clinical and Economic Review (ICER) has released a draft evidence report assessing the effectiveness and value of calcitonin gene-related peptide (CGRP) inhibitors in the prevention of migraine. The report included the budget impact of Amgen Inc. and Novartis AG’s Aimovig compared to no treatment or to that of Allergan plc’s Botox.


Pharmaceutical News

Inside the Beltway/State Regulatory Developments

The President will give a speech on drug prices next week, although he is not expected to unveil any new proposals. If you need a refresher on the Administration’s thoughts re drug pricing, this Health Affairs piece from Weekly View favorite (and Midwest CEPAC member) Rachel Sachs is a good place to start: Budget, White Paper Provide Insight Into Trump Administration’s Strategy On Drug Pricing.

Trump to give speech on lowering drug prices this month

President Trump Donald John Trump House Republicans push Mulvaney, Trump to rescind Gateway funds Pruitt spent K flying aides to Australia to prep for later-canceled visit: report Rosenstein told Trump he is not a target of Mueller probe: report MORE is scheduled to give a speech on prescription drug prices on April 26, the White House said Monday, as he calls for making the drugs more affordable.

Gottlieb talks about revamped processes and resources for drug reviews.

FDA to revamp drug reviews

FDA Commissioner Scott Gottlieb Wednesday described the agency’s plans to revamp the drug review process, including initiatives intended to make reviews more consistent and to support the creation of hundreds of product-specific drug development guidance documents. Gottlieb presented FDA’s plans for investing the $400 million FY19 funding boost the Trump administration has requested (see BioCentury, Feb.

Ned Sharpless, Director of the National Cancer Institute, discusses his priorities.

These are the top priorities for the nation’s top cancer doctor

A Federal Appeals Court struck down Maryland’s drug price law by a 2-1 margin overturning a lower court’s ruling. The reasoning of the dissenting opinion seems to indicate there may be more court activity ahead.

Federal Appeals Court Puts Chill On Maryland Law To Fight Drug Price-Gouging

This story also ran on This story can be republished for free ( NPR. details). States continue to battle budget-busting prices of prescription drugs. But a federal court decision could limit the weapons available to them – underscoring the challenge states face as they, in the absence of federal action, go one-on-one against the powerful drug industry.

Industry Trends

The Washington Post ran a piece about the pricing strategy of a drug being changed after researchers suggested that patients could use a lower dosage. Interestingly, Kevin Drum (no friend of pharma) wrote a piece defending the change and reminding us that no one expects the price of a drug to be based on the cost of materials.

Science hinted that cancer patients could take less of a $148,000-a-year drug. Its maker tripled the price of a pill

In which I defend a rapacious pharmaceutical company

The Washington Post reports on a group of doctors who were testing lower dosages of an expensive blood cancer drug called Imbruvica to see if it was effective at lower doses.

Authors suggest we should “limit the use of high-priced, marginally effective drugs.”

Defining Value in Targeted Cancer Drugs

Spending on cancer drugs in the United States has increased substantially over the past 5 years, from $26 billion in 2012 to more than $45 billion in 2016. Targeted cancer drugs, including small molecules, monoclonal antibodies, and other therapies based on genomic and related analyses, contributed 60% of this spending growth.

Harvard Pilgrim continues its interest in outcomes-based pricing deals, inking another one with AstraZeneca.

Pushing ‘value,’ Harvard Pilgrim tests outcomes deals

Harvard Pilgrim Health Care Inc. will pay less for AstraZeneca plc’s asthma drug Symbicort if its members experience worsening symptoms beyond a predetermined level set by an outcomes-based contract the two companies announced on April 18.

There are almost as many CAR-T trials going on in China as there are in the US.

As gene editing explodes, a new report from Goldman says Chinese groups are seizing the lead on CRISPR and CAR-T studies

Contrary to what you might believe, biopharma companies in the US are not the leaders in clinical trials using gene editing tech. While a slate of US/European pioneers have been lining up their first human trials, investigators in China are already well ahead in testing CRISPR-edited cells in humans, according

And China’s interest in biotech extends to our shores.

Chinese Money Floods U.S. Biotech as Beijing Chases New Cures

Effort to spur medical innovation sparks an influx of capital Investor hunger lets startups pick and choose their partners Chinese investors are pumping money into U.S. drug startups as Beijing seeks to become a global leader in new medicines, adding to a flood of cash flowing to groundbreaking health firms. Venture-capital funds based in China poured $1.4 billion into private U.S.

Amazon takes a small step back in its plans to sell drugs to hospitals – but still exploring many health care opportunities. The stock market reacted to the news: CVS Rallies After Amazon Report and Drug suppliers’ shares rise as looming Amazon threat appears to ease.

Amazon has shelved a plan to sell drugs to hospitals, and insiders say there are two reasons why

Amazon Business, which sells bulk items to business customers, has shelved its plan to sell and distribute pharmaceutical products after considering it last year, according to people familiar with the matter. Instead, the company is focused on selling less sensitive medical supplies to hospitals and smaller clinics through Amazon Business – and it has found that business to be more challenging than expected.

Walgreens and CVS are adding drug disposal kiosks to some stores. For many, the only way to dispose of unwanted and unneeded drugs has been the semi-annual drug take-back day. The next one is April 28th.

Drugstores are making it easier to empty your medicine cabinet

Walgreens started adding drug disposal units in 2016 and now has 600. It’s collected 155 tons of medications since the program began. AmerisourceBergen, the Blue Cross Blue Shield Association, Pfizer and Prime Therapeutics are partnering with Walgreens to add kiosks to another 900 stores.

Company/Drug Specific

On Thursday Allergan indicated they might bid for Shire, then hours later said never mind, they won’t be bidding. Meanwhile, Shire rejected Takeda’s initial bid of $60 billion, but indications are negotiations are ongoing.

Shire Open to Higher Takeda Offer After Rejecting $60 Billion

said it has turned down repeated takeover offers from , the latest for about $60 billion, and said the two sides are in talks about another “more attractive” transaction. Takeda said it offered 46.50 pounds ($66.16) a share in cash and stock last week for Shire in a bid to extend its global reach.

Sanofi to leave the generics business in $2.4 billion deal.

Sanofi to Sell Generics for $2.4 Billion to Focus on Biotech

In a different $2.4 billion deal, Shire sells its oncology unit to Servier.

Servier buys Shire’s oncology unit for $2.4B in the run-up to Takeda’s final buyout offer – if it makes one

If Takeda comes knocking on Shire’s door for what would be the largest acquisition in 2018, it won’t get the whole package it was angling for. Shire announced it’s selling its oncology business to French immuno-oncology company Servier for $2.4 billion. The deal comes after Takeda expressed interest for a

Impressive early results for gene therapy treatment of beta thalassemia (a genetic disorder in which the body does not make enough hemoglobin).

Gene therapy could free some people from a lifetime of blood transfusions

Joern Pollex | Getty A one-time, experimental treatment for an inherited blood disorder has shown dramatic results in a small study. It’s a major milestone for the company that created it, Bluebird Bio, and the latest in a string of successes for gene therapies, which aim to modify a person’s DNA to treat disease.

The first drug derived from marijuana received a unanimous recommendation for approval from the FDA advisory panel. The drug, unlike medical marijuana, does not make people high.

Panel recommends FDA approval of epilepsy drug derived from marijuana

n expert panel on Thursday unanimously recommended that the Food and Drug Administration approve a new medicine for two rare and devastating forms of epilepsy, paving the way for the authorization of what would be the country’s first medication made from marijuana.

Reasons for the FDA’s about-face may never be known.

In a stunning about face, the FDA is now welcoming the new drug application from Alkermes it rejected 2 weeks ago

Somebody at the FDA just got a rude wake up call. Just two weeks after the FDA invited Wall Street to pummel Alkermes $ALKS by refusing to even accept its applications for the depression drug ALKS-5461, the biotech says that the agency has suddenly decided to simply change its mind.

Weekly View In-depth

Lung cancer, immunotherapy, and checkpoint inhibitors, oh my!

On Monday at the annual meeting of the American Association for Cancer Research, trial results were released for studies combining checkpoint inhibitors and chemotherapy for lung cancer. Overall results were positive. While it is fair to worry that immunotherapies have been overhyped, and it is unquestionable that there is a sizeable population that is not being helped by them, it is also clear that for lung cancer they now are the state of the art for care with real survival benefits. ICER has taken an in-depth look at the topic.

First an overview of Monday’s results.

I/O combos inch forward in pharma’s lung cancer quest

Combining immunotherapy with other drugsmay become a more common choice as an initial treatment for lung cancer, bolstered by positive results from two major clinical studies testing checkpoint inhibitors made by Merck & Co. and Bristol-Myers Squibb Co.

Next, more details on Keytruda (the one some are saying is the “winner”).

Keytruda plus chemo dramatically improves lung cancer patients’ survival

ewly diagnosed lung cancer patients are half as likely to die of the disease if they take a Merck drug alongside standard chemotherapy, according to new trial data that eclipsed doctors’ expectations. The trial, which compared a combination of chemo and Merck’s blockbuster Keytruda against chemo alone, found that patients in the combo group were 51 percent more likely to be alive after one year.

While Opdivo had positive results, they were not nearly as good as Keytruda’s. Wall Street reacted accordingly.

Coke Versus Pepsi of Drugmakers? Wall Street Doesn’t Buy It

Hours after markets closed on Bristol-Myers Squibb Co.’s worst day in more than a year, executives used a sugary analogy to explain to Wall Street why the drugmaker’s new cancer treatment would do just fine against its main competitor. “Point blank, these are Coke and Pepsi in lung cancer, OK?”

But it wasn’t all bad news for Bristol-Myers and Opdivo as the Opdivo-Yervoy combination received approval for first-line kidney cancer treatment.

Bristol-Myers’ Opdivo-Yervoy duo snares a quick FDA nod in first-line kidney cancer

After an early setback for Bristol-Myers Squibb’s Opdivo-Yervoy combo in first-line kidney cancer-when the duo failed to stave off kidney cancer progression better than Pfizer’s Sutent did-the combo pressed on to show it could help patients live longer. And now, the New Jersey drugmaker has the kidney cancer go-ahead it’s been chasing.

Also this week in lung cancer news.

AstraZeneca wins expanded OK to use Tagrisso in frontline lung cancer niche, spurring hope of hitting $3B sales goal

The FDA has come through with a speedy approval for AstraZeneca’s standout cancer drug Tagrisso as a frontline treatment for EGFR-mutated non-small cell lung cancer. The OK, just four months after the agency offered a priority review, marks another big step forward toward AstraZeneca’s goal of gaining a larger share

 

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